Advancing Innovation in Rare Disease Research
2025-06-02 08:00 - 2025-06-03 17:00 (America/Vancouver)
Join researchers, clinicians, and industry leaders to discuss real-world research breakthroughs, novel #ClinicalTrial designs, and how NORD’s Rare Disease Centers of Excellence are leading collaborative efforts in #RareDisease innovation.

Webinar - Key Considerations for Including Pediatric Populations with Neurodevelopmental Conditions in Clinical Trials
2025-06-18 12:00 - 13:00 (America/Edmonton)
Speakers: Evdokia Anagnostou and Mayada Elsabbagh Title: Key considerations for including pediatric populations with neurodevelopmental conditions in clinical trials

Defeat Duchenne Canada-Industry Partner Update: REGENXBIO
2025-06-23 16:00 - 17:00 (America/Edmonton)
Join Defeat Duchenne Canada for an educational webinar featuring Dr. Jahannaz Dastgir, Executive Director of Clinical Development at REGENXBIO, as she shares the latest updates on RGX-202, an investigational gene therapy for the treatment of Duchenne muscular dystrophy, and their Phase 1/2/3 AFFINITY DUCHENNE® trial.
This session will provide valuable insights into the science behind RGX-202, trial progress, and what this potential investigational treatment could mean for the Duchenne community. Whether you are a parent, caregiver, clinician, or advocate, this webinar is an opportunity to stay informed and ask questions directly to the team leading this important research.

THE FESTIVAL OF GENOMICS & BIODATA Boston
2025-06-24 08:00 - 2025-06-25 17:00 (America/New_York)
The Festival is designed principally for scientists and clinicians who are working in the fields of genomics, other omics, and/or using biodata , to further research, drug discovery, healthcare and – ultimately – deliver better patient outcomes.

Topics covered include genomics, single cell/spatial biology, multiomics, biodata, cancer research, drug discovery, AI, microbiome, proteomics, liquid biopsy and so much more.

2nd Global Cell & Gene Therapy Summit
2025-07-07 08:00 - 2025-07-09 17:00 (America/New_York)

IMPaCT Webinar - An Overview of Current and Future Clinical Trials in Children: a European Perspective
2025-07-21 12:00 - 13:00 (America/Toronto)
Dr. Eva Degraeuwe (MD-PhD) is a dedicated pediatric resident and researcher in pediatric nephrology and global clinical trial optimilisation. Working in many Global networks focused on rare and low prevalance diseases, she has completed her PhD in 2024 participating and guiding over 35 clinical trials. Moreover, she has a passion for patient engagement, especially within a clinical trial setting.
Webinar Overview:
– European network setting and the role of networks
– Pathways to clinical trial optimilisation
– Patient engagement and what has been achieved

6th International Hypothalamic Hamartoma Symposium
2025-09-04 08:00 - 2025-09-05 17:00 (America/Belize)
Presented by Hope for HH and Hospital Sant Joan de Déu Barcelona, this symposium will gather leading experts, patients, and caregivers from around the world to share the latest research, clinical advances, and treatment strategies for Hypothalamic Hamartoma (HH)! Alexis Arzimanoglou and Helen Cross are part of the Symposium Committee.

Rare Trials Summit
2025-09-09 08:00 - 2025-09-10 17:00 (America/Edmonton)
Bringing together key stakeholders in the clinical research community to accelerate the development of new rare disease therapies from average 10-12 years to under 3 years.

IMPaCT Webinar: Lessons Learned from the Innovative Pediatric Clinical Trials (iPCT) Network
2025-09-10 10:00 - 11:00 (America/Edmonton)
Speakers: Tannis Erickson & Naveen Poonai Title: Lessons Learned from the Innovative Pediatric Clinical Trials (iPCT) Network

2025 NMD4C Annual Meeting
2025-09-11 08:00 - 17:00 (America/Toronto)
Registration is now open for the 2025 NMD4C Annual Meeting!
Join NMD4C in Ottawa on Thursday, September 11, for a full day of dynamic discussions, research updates, and networking opportunities—just ahead of the 7th Ottawa International Conference on Neuromuscular Disease & Biology.

Ottawa NMD 2025 Conference
2025-09-11 08:00 - 2025-09-13 17:00 (America/Toronto)
7th Ottawa International Conference on Neuromuscular Disease and Biology will take place at the Fairmont Château Laurier in Ottawa, Canada on September 11-13, 2025.
The Ottawa NMD 2025 conference will offer an outstanding program emphasizing recent breakthroughs in basic and translational research and clinical discoveries in neuromuscular disease.

Undiagnosed Hackathon 2025
2025-09-21 08:00 - 2025-09-24 17:00 (America/Chicago)
350 million people live with an undiagnosed disease worldwide. Genome sequencing can provide answers for 40% of the People Living With Undiagnosed Disease (PLWUD) but around 60% still don’t receive a diagnosis. The purpose of the Undiagnosed Hackathon is trying to find new ways and collaborations to solve the undiagnosed diseases, that can’t be solved today. In this unique event multidisciplinary teams of clinicians, bioinformaticians/molecular biologists, scientists, developers, AI specialists and brilliant minds will work together to tackle the most difficult diseases.

6th Western Canadian Neuromuscular Conference
2025-09-25 08:00 - 2025-09-27 17:00 (America/Edmonton)
The 6th Western Canadian Neuromuscular
Conference is confirmed for 2026! Don’t miss
the opportunity to network with leading experts
and stay at the forefront of medical
advancements in the field of neuromuscular
diseases.

Canadian Rare Disease Symposium: Preparing for Clinical Trials – International Regulatory Insights, and cGMP Gene Therapy Manufacturing capabilities in Canada
2025-10-01 13:00 - 16:00 (America/Toronto)
The Stem Cell Network (SCN) and the National Research Council Canada (NRC) are excited to contribute to the national conversation on cell and gene therapy for rare diseases. Building on the 2024 event titled “Canadian Rare Disease Ecosystem Symposium: Genetic Therapies for Rare Disease”, SCN and NRC are pleased to invite you to the Canadian Rare Disease Symposium: Preparing for Clinical Trials – International Regulatory Insights, and cGMP Gene Therapy Manufacturing Capabilities in Canada–a focused half-day event dedicated to advancing gene and cell therapy development in Canada through regulatory alignment and manufacturing readiness.
This virtual symposium will convene international regulatory authorities, Canadian manufacturing experts, and rare disease developers to explore two critical enablers of translational success: clinical trial readiness and cGMP manufacturing capacity.

CAGC & CCMG Joint Conference
2025-10-05 08:00 - 2025-10-08 17:00 (America/Edmonton)
The CAGC & CCMG Joint Conference will be a unique gathering of medical and laboratory geneticists and genetic counsellors with diverse expertise, all sharing a common interest in education and advocating for best practices in clinical and laboratory genetics. Through presentations, workshops, breakout sessions, abstract sessions, and engagement with industry partners, the event propels forward clinical practice, scientific advancements, and excellence across all aspects of genetics in Canada.

30th Annual International Congress of the World Muscle Society
2025-10-07 08:00 - 2025-10-11 17:00 (Europe/Vienna)
The World Muscle Society is a dynamic community that aims to promote, disseminate, and share all aspects of neuromuscular physiology and diseases, from basic science to patient care. It encompasses a broad range of scientists and healthcare professionals who share the common interest for neuromuscular disorders.

The World Orphan Drug Congress
2025-10-27 08:00 - 2025-10-29 17:00 (Europe/Amsterdam)
The World Orphan Drug Congress is the largest and most established orphan drug & rare disease meeting of its kind across the globe.
From cell and gene therapy, genetic testing, and market access, to real world evidence, this one meeting covers the whole orphan drugs value chain where science, government and manufacturers all come together to create groundbreaking progress.

Cardiac Precision Medicine in the 21st Century
2025-10-29 08:00 - 2025-10-31 17:00 (America/Toronto)
The 10th International Cardiovascular Genomic Medicine & SickKids Cardiac Precision Medicine Conference is making its debut in North America! This year’s theme, “Cardiac Precision Medicine in the 21st Century,” delves into cardiovascular genomics and precision medicine in the era of ‘Omics’, AI and digital medicine. Join us as we bring together international experts and innovators to explore transformative research and collaborative opportunities that can shape the future of cardiovascular medicine.
This event will feature the 4th William Harvey Oration delivered by Dr. Sharlene Day and the Dr. Michael Braudo Lectureship delivered by Dr. Ma’n H. Zawati.

Easing Pain, Advancing Care: A New Pathway for Children with Severe Neurological Impairments
2025-10-29 09:00 - 10:00 (America/Edmonton)
This webinar will provide an overview of the multi-phased project, from clinical trial to implementation. It will highlight the clinical complexity of assessing pain and irritability; the project’s relevance and impact for clinicians, children with health complexity, and their families; as well as key lessons for patient-oriented research and implementation science.

ITINERARE Symposium 2025
2025-11-18 08:00 - 17:00 (Europe/Zurich)
The programme features experts from a variety of fields, ranging from basic and translational research to clinical application and the ethical, social and legal aspects of rare disease research.

2025 IMPaCT Summit
2025-11-18 08:00 - 2025-11-19 17:00 (America/New_York)

2nd International Conference on Clinical Research Networks (CRNs) for Rare Diseases
2025-12-09 08:00 - 2025-12-10 17:00 (America/Edmonton)
This second edition will convene a wide range of international experts, including researchers, clinicians, patient advocates, industry representatives, policymakers, and funders to strengthen the collaborative ecosystem that underpins clinical research in rare diseases worldwide.

Every Relapse Matters: The Evolving NMOSD & MOGAD Treatment Landscape in Canada
2025-12-11 17:30 - 18:30 (America/Edmonton)
The Sumaira Foundation Canada is pleased to invite you to an important educational webinar on Thursday, December 11th at 7:30 PM EST. This session will explore the evolving treatment options for individuals living with AQP4+ NMOSD, seronegative NMOSD, and MOGAD in Canada.

Webinar: Lymphodepletion for CAR-T/IEC Outcomes
2025-12-17 12:00 - 14:00 (America/Toronto)
Achieving consistent and effective outcomes in CAR T and other immune effector cell (IEC) therapies relies heavily on getting lymphodepletion “right.” Emerging evidence shows that both under- and over-exposure can impact safety, efficacy, and long-term persistence, making optimized dosing strategies increasingly critical as the field evolves.
This webinar will explore how model-informed and data-driven approaches can help identify the optimal lymphodepletion exposure window, improve predictability, and support more standardized practices across patient populations and treatment centers. Speakers will discuss current challenges, scientific rationale, clinical insights, and practical considerations for implementation.

RealiseD Webinar 1: Realising Clinical trials in Ultra-Rare diseases
2026-01-13 17:00 - 18:30 (America/Edmonton)
Join RealiseD’s first webinar to learn how RealiseD is pioneering a collaborative and comprehensive approach to clinical trials in ultra-rare diseases. During the webinar, they’ll walk you through the project’s objectives, structure, and methodologies—and show how you can be part of this transformative journey.

RealiseD Webinar 2: Evidence Assessment Framework. A need to change mindset from developers, regulators and HTA
2026-01-20 17:00 - 18:30 (America/Edmonton)
The second webinar turns the spotlight on one of the most critical issues in rare disease research: the generation and assessment of evidence. François Meyer, a leading consultant in health technology assessment, will examine why current frameworks often fail to deliver predictable and aligned outcomes, resulting in delays and inequalities in patient access. The session will explore the global and European initiatives aimed at addressing these shortcomings and present the RealiseD consortium’s efforts to advance scientific and operational solutions. More importantly, it will advocate for a fundamental shift in mindset—one that embraces flexibility and innovation in the way evidence is generated, interpreted, and assessed.

CMT episode 3: Bioarkers and outcome measures for clinical trial readiness in CMT
2026-01-22 08:00 - 09:00 (America/Edmonton)
Delivered by Prof. Mary Reilly (University College London, Department of Neuromuscular Diseases, UK), Thu 22 Jan 2026 at 17:00 Paris Time This is a EURO-NMD webinar in collaboration with ERN-RND (rare neurological disorders) and European Academy of Neurology.

CATALIS-Clinical Research 101: Webinar 1
2026-01-22 18:00 - 18:30 (America/Toronto)
Webinar 1 on January 22, 2026 from 6 pm to 6:30 pm

Canada's Drug Agency-Preconsultation Webinar: Improvements to the Drug Reimbursement Review Process
2026-01-28 14:00 - 15:30 (America/Toronto)
Join CDA’s preconsultation webinar on proposed improvements and upcoming procedural changes to our drug reimbursement review process.
You will hear from their senior leaders how these plans will advance our efforts to modernize and streamline drug reimbursement reviews.

CATALIS-Clinical Research 101: Webinar 2
2026-01-29 18:00 - 18:30 (America/Toronto)
Webinar 2 on January 29, 2026 from 6 pm to 6:30 pm
